Navigation Links
Repeated hUCB injections may improve prognosis of children with deadly inherited disorder

Putnam Valley, NY. (Mar. 28 2014) New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.

In a recent study into the effects of human umbilical cord blood mononuclear cells (hUCB MNCs) when they are injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B), researchers found that repeated injections into laboratory mice modeled with the disorder had clear benefits for the mice receiving multiple injections over control groups that received single injections of either a high or low dose of cells.

The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at:

MPS III B results from a genetically programmed deficit of the Naglu enzyme. The deficit creates a build-up of heparan sulfate - a complex carbohydrate - that accumulates in lysosomes, cells that are responsible for waste disposal. With MPS III B, accumulations of heparin sulfate in the tissues are not eliminated and the accumulation causes damage to multiple organs, including the brain.

"Cell therapy has recently received attention as a potential treatment for lysosomal storage diseases," said study lead author Dr. Allison E. Willing, of the Center of Excellence for Aging and Brain Repair in the Morsani College of Medicine at the University of South Florida. "We have previously shown that a single hUCB injection into the cerebral ventricle of pre-symptomatic mice, or intravenous cell delivery at different disease stages, had a beneficial effect on the enzyme deficient mice. In the current study, we examined whether administering repeated doses of hUCB MNCs would have a greater effect than a single dose and help to prevent progressive neurodegeneration."

Using three groups of mice modeled with Naglu deficiency by knocking out the Naglu enzyme, the researchers injected one group with repeated doses of hUCB MNCs over a six month period. They administered single doses - either high or low doses - to two other groups of similarly modeled mice. The group that had repeated hUCB MNC doses demonstrated a variety of favorable benefits.

To determine the benefits of repeated hUCB MNC injections, the researchers measured several behavioral and clinical outcomes before and after six months of treatment. These included anxiety, levels of heparin sulfate accumulation, and subsequent pathology in various anatomical brain locations.

"Repeated injections of hUCB MNCs produced the greatest neuroprotection," stated study co-lead author Dr. Svitlana Garbuzova-Davis, of the Center of Excellence for Aging and Brain Repair in the Morsani College of Medicine at the University of South Florida. "Hippocampal structural architecture remained intact in the repeated dose-treated mice as compared to the other groups. Also, there was restoration of the dendritic tree in the group receiving repeated doses. We also saw a striking reduction in microgliosis and microglial activation after hUCB MNC treatment."

The researchers speculated that administering hUCB MNCs may decrease neuropathy through modulation of inflammatory and immune processes as the hUCB MNCs produced numerous neurotrophic and growth factors.

"We demonstrated that hUCB MNCs were particularly effective at modulating anxiety in the Naglu knockout mice,"concluded Paul R. Sanberg, distinguished professor at USF and principal investigator of the Children's Medical Research Foundation funded project. "Our results suggest that repeated administrations of hUCB MNCs produce greater amelioration of the underlying disease pathology. However, further studies will be necessary to determine if this treatment regimen can slow the progression of the disease, increase survival while minimizing symptoms, and determine whether improved outcomes are a function of enzyme administration, decreased inflammation, or both."

"This study highlights the benefits of using multiple injections rather than a single injection to treat Sanfilippo syndrome type III B" said Dr. John Sladek, Cell Transplantation section editor and professor of neurology and pediatrics at the University of Colorado School of Medicine. "This use of multiple treatments may be applicable to other neurodegenerative disorders, as already suggested by animal studies published by the same group for the treatment of amyotrophic lateral sclerosis."

Contact: Robert Miranda
Cell Transplantation Center of Excellence for Aging and Brain Repair

Related medicine news :

1. Repeated exposure to traumatic images may be harmful to health
2. Study: Repeated surgeries appear to extend life of patients with deadliest of brain cancers
3. Breast cancer risk estimates increased with repeated prior ct and nuclear imaging
4. No need for routine repeated CT scans after mild head trauma, reports neurosurgery
5. Facial dog bites in children may require repeated plastic surgery
6. Necrosis after cortisone injections
7. Reused Vials, Unsafe Injections Threatening Patients: CDC
8. US Vitamin Injections Looks Into B12 Shots for Fitness in New Article
9. US HCG Injections Posts New Article about the Spartacus Workout
10. US HCG Injections Posts Inspirational HCG Diet Customer Success Story
11. hCGTreatments / Diet Doc hCG Diets & Weight Loss Plans Announces New Alternative to Stomach Botox Injections
Post Your Comments:
(Date:11/25/2015)... ... November 25, 2015 , ... On November 10, 2015, Bohrer Brady, ... of Connecticut on behalf of a home health care worker who provided companionship services ... home health care workers employed by Humana, Inc., Humana at Home, Inc., and SeniorBridge ...
(Date:11/25/2015)... Canadian, OK (PRWEB) , ... November 25, 2015 , ... ... and International training center for the Narconon network, announced the release of a new ... Since 1966 the Narconon organization has been working with drug- and alcohol-addicted individuals with ...
(Date:11/25/2015)... , ... November 25, 2015 , ... ... double board certified facial plastic surgeon specializing in both surgical and non-surgical treatments, ... The Skin Spa at Hobgood Facial Plastic Surgery. , Highly trained and ...
(Date:11/25/2015)... ... ... Today, Mothers Against Drunk Driving (MADD) learned that the number ... first time since 2011. In 2014, there were 9,967 fatalities involving an alcohol impaired ... Highway Traffic Safety Administration (NHTSA), 32,675 people were killed in traffic crashes in 2014. ...
(Date:11/25/2015)... ... ... An unlikely combination of recycled plastic shopping bags in ... to have a more dignified and comfortable night’s sleep. , Residents of Friendship ... bags into sleeping mats for the homeless. The project, according to Jeannette ...
Breaking Medicine News(10 mins):
(Date:11/25/2015)... - Will Also Offer Point -of- ... --> - Will Also Offer Point ... --> - Will Also Offer ... (CME) Elsevier , a world-leading provider of ... diagnostic imaging textbooks and decision support tools, as well as prominent ...
(Date:11/24/2015)... , November 25, 2015 ... due to repeated failure of IVF cycles. After failure ... was totally dejected and had lost all hopes that she would be able ... Indian miracle child conceived after failure of over 15 ... abroad (UK) before they decided to take one last attempt with ...
(Date:11/24/2015)... Nov. 24, 2015  Array BioPharma Inc. (Nasdaq: ... Executive Officer, Ron Squarer , will present ... New York.  The public is welcome to participate ... Array BioPharma website.Event:Piper Jaffray Annual Healthcare ConferencePresenter:  , ... 2, 2015Time:1:30 p.m. Eastern Time Webcast: ...
Breaking Medicine Technology: